Summit Therapeutics reports positive data on its DMD treatment

Treatment with ezutromid reduced muscle damage by 23%

Summit Therapeutics reports positive DMD data,
Summit Therapeutics reports positive DMD data

Summit Therapeutics plc (SMMT), the drug discovery and development company advancing therapies for rare diseases and infectious diseases, today announces positive 24-week interim results from the open-label Phase 2 proof of concept clinical trial, PhaseOut DMD.

PhaseOut DMD is evaluating the utrophin modulator ezutromid in patients with Duchenne muscular dystrophy (‘DMD’). The focus of the planned interim analysis was on biopsy measures that show:

  • Treatment with ezutromid resulted in a statistically significant and meaningful reduction in muscle damage as measured by a 23% decrease in mean developmental myosin in muscle biopsies at 24 weeks compared to baseline. Developmental myosin is a biomarker of muscle damage and is found in repairing fibres.
  • A total of 14 of 22 patients showed a decrease in developmental myosin, with five of those showing a greater than 40% reduction.
  • Increase in mean utrophin protein intensity levels of 7% in biopsies at 24 weeks compared to baseline.

The combination of reduced muscle fibre damage and increased levels of utrophin provides the first evidence of ezutromid target engagement and proof of mechanism.

DMD is caused by genetic faults that prevent muscle cells from making dystrophin, a protein that maintains the structure and healthy functioning of muscles.

The absence of dystrophin, as seen in patients with DMD, leads to a catastrophic cycle of muscle damage and repair.

Utrophin protein performs a similar role to dystrophin in developing and repairing muscle fibres.

As a muscle fibre matures, utrophin is switched off and replaced by dystrophin in the case of healthy individuals. During the early stages of natural muscle repair, utrophin and developmental myosin are expressed concurrently, and are then slowly switched off.

Ezutromid aims to maintain utrophin expression in patients with DMD so it can substitute for the lack of dystrophin and break this cycle.

SMMT closed at $12.21, it last traded at $15.25.


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